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Molecular Therapy. Nucleic Acids|September 14, 2016
Reprogramming the Dynamin 2 mRNA by Spliceosome-mediated RNA Trans-splicingDelphine Trochet, Bernard Prudhon, Arnaud Jollet, et al.
Nature Structural & Molecular Biology|December 28, 2010
Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAsVirginie François, Arnaud F Klein, Cyriaque Beley, et al.
Nucleic Acids Research|July 18, 2013
Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approachesStéphanie Lorain, Cécile Peccate, Maëva Le Hir, et al.
Molecular Therapy. Nucleic Acids|March 4, 2018
Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular DystrophyFeriel Azibani, Astrid Brull, Ludovic Arandel, et al.
Disease Models & Mechanisms|February 12, 2017
Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compoundsLudovic Arandel, Micaela Polay Espinoza, Magdalena Matloka, et al.
Nature Communications|May 29, 2015
Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophyFrédérique Rau, Jeanne Lainé, Laetitita Ramanoudjame, et al.
The Journal of Allergy and Clinical Immunology|December 17, 2013
Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndromeNiek P van Til, Roya Sarwari, Trudi P Visser, et al.
Nature Communications|April 12, 2016
Splicing misregulation of SCN5A contributes to cardiac-conduction delay and heart arrhythmia in myotonic dystrophyFernande Freyermuth, Frédérique Rau, Yosuke Kokunai, et al.
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